Single-center randomized phase 2 trial of neoadjuvant and adjuvant toripalimab for locoregionally advanced nasopharyngeal carcinoma

Patients with locoregionally advanced nasopharyngeal carcinoma with a high pretreatment plasma concentration of Epstein-Barr virus DNA are at high risk for recurrence after concurrent chemoradiotherapy (CRT). A randomized phase 2 trial at Sun Yat-sen University (Guangzhou, China) evaluated neoadjuvant and adjuvant toripalimab (anti-PD-1) for these patients. Patients were randomized to neoadjuvant toripalimab or placebo, followed by concurrent CRT and adjuvant toripalimab or placebo. Two-year progression-free survival rate was 92.0% in the toripalimab group versus 74.0% in de placebo group (sHR 0.40; p=0.019). The safety profile was manageable. The authors conclude that the sandwich approach involving toripalimab in the neoadjuvant and adjuvant phases combined with concurrent CRT could be a highly promising therapy for locoregionally advanced nasopharygeal carcinoma.